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5 advancements in rare disease research over the past year

10 biotech companies making a difference in rare diseases

Beyond Biotech podcast 35: Rare Disease Day

Moderna and Life Edit Therapeutics look to speed up gene editing therapies

First patient enrolled in Pharming APSD trial  

FDA approves alpha-mannosidosis drug

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Positive results for INOVIO recurrent respiratory papillomatosis trial

Janssen reports positive topline phase 2 HDFN results

Sponsored by 2 Feb 2023

Improving rare disease diagnosis and treatment with next generation sequencing technology

Hundreds of genetic discoveries from FinnGen study 

In Depth 11 Jan 2023

Drug repurposing emerges as viable option for rare disease treatment

Opinion 12 Dec 2022

Artificial intelligence: a new era in rare genetic disease diagnosis

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